Aeterna Zentaris Receives Agreement from FDA on Special Protocol Assessment for Solorel® (AEZS-130, Macimorelin) to Complete Registration Study for Diagnosis of Adult Growth Hormone Deficiency
QUÉBEC, Dec. 20 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS, TSX: AEZ) (the "Company") today announced that it has reached agreement with the Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for Solorel® (AEZS-130, macimorelin) which will enable the Company to complete the ongoing registration study required to gain approval as a diagnostic test for Adult Growth Hormone Deficiency (AGHD).
"We are pleased with the agreement with the FDA and now look forward to the completion of the Phase 3 trial with Solorel® and the NDA filing in 2011 for use as a diagnostic test in AGHD", stated Juergen Engel, Ph.D., President and CEO at Aeterna Zentaris. "In line with our innovative approach, Solorel® could become the first approved oral test for the diagnosis of AGHD, providing patients with a potentially safer, accurate and more convenient alternative to the current injectable tests."
The study was initiated to compare the performance of Solorel® against the then-available diagnostic growth hormone releasing hormone (GHRH) Geref Diagnostic® + Arginine (ARG) standard test. GHRH Geref Diagnostic® was subsequently withdrawn from the market, worldwide, in 2008. At the time of withdrawal, the trial included 42 patients with AGHD and ten control subjects. In 2009, Aeterna Zentaris regained the rights to Solorel® and had been working with the FDA to establish the best way forward to complete this registration study and continue to utilize the data obtained thus far, in light of the loss of the original comparator.
The protocol is titled,"A Multi-Center Study Investigating a New, Oral Growth Hormone Secretagogue (AEZS-130, formerly ARD-07) as a Growth Hormone (GH) Stimulation Test in Terms of Safety and Efficacy." (Clinicaltrials.gov Identifier: NCT00448747)
The SPA agreement has resulted in a modification of the original study, but does not alter the basic study design so that the completed portion of the study will work with the new part of the study to provide one, complete, Phase 3 study.
The completed part of the study was a two-way crossover study, and included 42 patients with confirmed AGHD or multiple pituitary hormone deficiencies and a low insulin-like growth factor-I. A control group of 10 subjects without AGHD were matched to patients for age, gender, body mass index and (for females) estrogen status.
Each patient received two dosing regimens in random order, while fasting, at least 1 week apart. One regimen consisted of a 1 µg/kg (max. 100 µg) dose of GHRH (Geref Diagnostic®, Serono) with 30 g of ARG (Ar-Gine®, Pfizer) administered intravenously over 30 minutes; the other regimen was a dose of 0.5 mg/kg body weight of Solorel® given in an oral solution of 0.5 mg/ml.
SPA Agreed Study Amendment
Completion of the study will be accomplished with the following revisions/additions to the current protocol:
- An additional 30 normal controls subjects will be enrolled to match the AGHD patients from the original cohort;
- Further, an additional 20 subjects will be enrolled - 10 AGHD patients and 10 matched normal control subjects;
- The above will bring the database to approximately 100 patients;
- All subjects will be receiving a dose of 0.5 mg/kg body weight of Solorel®;
- As a secondary endpoint, the protocol will require that at least 8 of the 10 newly enrolled AGHD patients be correctly classified by a pre-specified peak Growth Hormone (GH) threshold level.
Solorel®, a ghrelin agonist, is a novel orally active small molecule that stimulates the secretion of growth hormone (GH).The orally-administered product is currently in a Phase 3 trial for use as a simple diagnostic test for adult growth hormone deficiency (AGHD). Solorel® works by stimulating a patient's GH secretion after which a healthcare provider will measure how well the body responds to that stimulation based on the patient's GH levels over a period of time. Low GH levels, despite giving an effective stimulating agent, confirm a diagnosis of AGHD. Solorel® has been granted orphan-drug designation by the FDA for use as a diagnostic test for growth hormone deficiency. Aeterna Zentaris owns the worldwide rights to Solorel® which, if approved, would become the first orally available diagnostic test for AGHD.
AGHD affects 35,000 adult Americans, with 6,000 new adult patients diagnosed each year. Growth hormone (GH) not only plays an important role in growth from childhood to adulthood, but helps promote good health throughout life. AGHD is usually characterized by low energy levels, decreased strength and exercise tolerance, increased weight or difficulty losing weight, emotional changes, anxiety and impaired sleep. Available diagnostic tests for AGHD are complex and can produce significant side effects.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a late-stage oncology drug development company currently investigating potential treatments for various cancers including colorectal, ovarian, endometrial cancer and multiple myeloma. The Company's innovative approach of "personalized medicine" means tailoring treatments to a patient's specific condition and to unmet medical needs. Aeterna Zentaris' deep pipeline is drawn from its proprietary discovery unit providing the Company with constant and long-term access to state-of-the-art therapeutic options. For more information please visit www.aezsinc.com.